FDA授予TaiMed Biologics（中裕新药）的HIV单克隆抗体、长效抗逆转录病毒药物Ibalizumab优先审评资格

• 生物制剂许可证申请（BLA）接受优先审评，目标行动日期为2018年1月3日

• Ibalizumab扩展接收计划（Expanded Access Program，EAP）目前招募新患者

加拿大蒙特利尔 2017年6月30日：Theratechnologies（TST：TH）今天宣布得到其合作伙伴TaiMed Biologics，Inc.（中裕新药）的通知，美国食品药品监督管理局（FDA）已经接受对中裕新药的Ibalizumab的生物制剂许可证申请（BLA）的优先审评，该药物申请的适应症是1型多药耐药性人类免疫缺陷病毒（multidrug resistant Human Immunodeficiency Virus-1，MDR HIV-1）。如果获得批准，Ibalizumab单抗将成为近10年内引入崭新作用机制的第一个抗逆转录病毒治疗药物（antiretroviral treatment，ART），唯一一款不需要每日给药的疗法。

“我们很高兴离获批又靠近了一步，从而能够向病人提供一个具有崭新作用机制的重要的新疗法，这些病人体内的病毒已经对很多类药物产生耐药，并且病情的长期治疗的选择有限。” Theratechnologies公司总裁兼首席执行官Luc Tanguay先生表示，“授予优先审评资格非常重要，如果批准，Ibalizumab单抗对治疗这种严重疾病将有重大改善。”

FDA已经为Ibalizumab单抗的申请设定了处方药用户费用法案（PDUFA）目标日期为2018年1月3日。优先审评资格将加速FDA的审评，审评时间从接受之日起的10个月减少到6个月。此外，FDA在2015年授予Ibalizumab“突破疗法认定”，该认定授予可能比目前已有疗法显着改善对严重的、危及生命的疾病的治疗的药物。FDA还在2014年授予该药物“孤儿药资格”。

中裕新药基于TMB-301的3期临床研究的数据于2017年5月3日提交BLA，该研究是单臂试验，历时24周，研究Ibalizumab加上一款优化背景疗法（optimized background regimen，OBR）治疗之前接受过其它疗法的患者，这些患者产生耐药，病情恶化。

Ibalizumab单抗扩大接收计划（EAP），TMB-311临床研究，正在进行中并招收患者。有关TMB-311（NCT02707861）的更多信息，请参阅ClinicalTrials.gov网站（www.clinicaltrials.gov）或该药物的研究网站（www.ibalizumab-eap.com）。

随着艾滋病病毒在体内的繁殖，病毒可能会突变以产生耐药菌株。病毒突变可能意味着以前控制病毒的艾滋病药物不再有效，导致治疗失败。在美国，有大约20,000至25,000名HIV-1携带者对三种不同类型的抗逆转录病毒药物中的至少一种具有耐药性。 这些患者中有多达12,000例在48周的治疗期间里治疗失败，需要他们的医生修改治疗方案。

关于Ibalizumab（伊巴珠单抗）

Ibalizumab单抗是正在被开发的用于治疗MDR HIV-1感染的研究性人源化单克隆抗体。与其它抗逆转录病毒药物不同，Ibalizumab单抗主要与CD4+ T细胞受体的第二细胞外结构域结合，远离主要组织相容性复合物II分子结合位点。 它可以防止艾滋病毒感染CD4+ 免疫细胞，同时保持正常的免疫功能。

伊巴珠单抗对所有获批的抗逆转录病毒药物产生耐药的HIV-1病毒有效。

关于 Theratechnologies

Theratechnologies（TSX：TH）是一家专业制药公司，致力于未满足的医疗需求，以促进滋病患者健康变老和提高他们的生活质量。有关Theratechnologies的更多信息，请访问公司网站www.theratech.com和SEDAR www.sedar.com。

资料来源：Theratechnologies

延伸阅读：

EAP临床试验（Expanded Access Program）

EAP临床试验是指制药企业为了让患有严重疾病且不适合参加对照试验的患者，在特定的条件下，能够得到正处于临床试验阶段的研究新药的治疗，而开展的一类临床试验。绝大部分的新药临床试验采用对照的设计以评估新药的安全性和有效性。来自这些临床试验的数据通常可被用于决定该药是否安全有效，并作为药物上市申请的基本依据。但有时，病人由于自身健康状况，年龄及其他因素不符合参加这些对照试验的条件，或其他原因不能被入选（例如，病人居住地距离临床研究中心过远）。这些患有严重疾病的患者有可能能从新药的治疗中获益，但却不能参加该药的临床试验。为了使这一类的病人也能受益，FDA允许这类药物的生产企业向那些病人提供在特定条件下获得新药治疗的机会，称之为”扩展的途径”。

新闻稿原文

FDA Grants Priority Review to HIV Monoclonal Antibody and Long-Acting Investigational Antiretroviral Ibalizumab

Biologics License Application (BLA) Accepted for Review with a Target Action Date of January 3, 2018

Ibalizumab Expanded Access Program (EAP) Currently Enrolling New Patients

MONTREAL, Canada I June 30, 2017 I Theratechnologies Inc. (Theratechnologies) (TSX:TH) today announced that it has been notified by its partner, TaiMed Biologics, Inc., that the U.S. Food and Drug Administration (FDA) has accepted for review the Biologics License Application (BLA) for ibalizumab as a treatment for multidrug resistant Human Immunodeficiency Virus-1 (MDR HIV-1). If approved, ibalizumab will be the first antiretroviral treatment (ART) with a new mechanism of action to be introduced in nearly 10 years and the only treatment that does not require daily dosing.

"We are excited to be one step closer to potentially bringing an important new treatment, with a new mechanism of action, to patients whose virus has become resistant to therapies in multiple classes and have limited treatment options for the long-term management of their condition," said Luc Tanguay, President and Chief Executive Officer, Theratechnologies Inc. "The granting of Priority Review status is important since it confirms that, if approved, ibalizumab would represent a significant improvement in the treatment of this serious condition," added Mr. Tanguay.

The FDA has set a Prescription Drug User Fee Act (PDUFA) target action date of January 3, 2018, for the ibalizumab application. Priority Review status accelerates FDA review time from 10 months to a goal of six months from the day of acceptance. In addition, ibalizumab received Breakthrough Therapy designation from the FDA in 2015, which is given if a therapy may provide a substantial improvement over what is currently available to address a serious and life-threatening condition. The FDA also granted Orphan Drug designation in 2014.

The BLA, submitted on May 3, 2017, is based on data from the phase III TMB-301 study, a single arm, 24-week study of ibalizumab plus an optimized background regimen (OBR) in treatment-experienced patients who had high pre-existing levels of drug resistance and advanced clinical disease.

The ibalizumab Expanded Access Program (EAP), or study TMB-311, is ongoing and enrolling patients. For more information about TMB-311 (NCT02707861), please refer to the ClinicalTrials.gov website (www.clinicaltrials.gov) or the study website (www.ibalizumab-eap.com).

As HIV multiplies in the body, the virus may mutate to produce drug-resistant strains. Viral mutations may mean that HIV medicines that previously controlled a person's virus are no longer effective, causing treatment to fail. There are approximately 20,000 to 25,000 Americans with HIV-1 that are resistant to at least one drug out of the three different classes of antiretroviral therapies. Up to 12,000 of these patients experience a virological failure over a period of 48 weeks of treatment, requiring their physician to modify their treatment.

About ibalizumab

Ibalizumab is an investigational humanized monoclonal antibody being developed for the treatment of MDR HIV-1 infection. Unlike other antiretroviral agents, ibalizumab binds primarily to the second extracellular domain of the CD4+ T cell receptor, away from major histocompatibility complex II molecule binding sites. It potentially prevents HIV from infecting CD4+ immune cells while preserving normal immunological function.

Ibalizumab is active against HIV-1 resistant to all approved antiretroviral agents.

About Theratechnologies

Theratechnologies (TSX:TH) is a specialty pharmaceutical company addressing unmet medical needs to promote healthy ageing and an improved quality of life among HIV patients. Further information about Theratechnologies is available on the Company's website at www.theratech.com and on SEDAR at www.sedar.com.

SOURCE: Theratechnologies

参考文献：

• 新闻稿：FDA Grants Priority Review to HIV Monoclonal Antibody and Long-Acting Investigational Antiretroviral Ibalizumab（点击阅读原文）

• 安卓健EAP临床试验方案公布（医药黑板报）

• Theratechnologies官网

• TaiMed官网

• FDA官网
  

• 其它网络信息

图片来源：参考文献及相关网站、网络

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